NEW YORK, NY – January 3, 2021 – Woolsey Pharmaceuticals, a repurposed therapeutics development company announced that the European Medicines Agency’s Committee for Orphan Medicinal Product has issued a positive opinion on the orphan drug application for the Company’s investigational drug, BRAVYL® (oral fasudil), for the treatment of Amyotrophic Lateral Sclerosis (ALS). The opinion will be forwarded to the European Commission for a final decision sometime in January.
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. ALS is part of a wider group of disorders known as motor neuron diseases.
There is a large and convincing body of preclinical evidence of BRAVYL’s effect on ALS in animal models. Additionally, there is persuasive human data from a compassionate use study in Europe, where three ALS patients were dosed with an intravenous version of BRAVYL for a month. In all three patients, decline in function was attenuated during and one month after fasudil infusions, in one patient breathing capacity increased during treatment, and in one muscle strength increased during treatment.
European orphan drug designation is granted to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that impact fewer than 5 in 10,000 patients in the European Union. Orphan drug designation gives companies certain benefits, including reduced regulatory fees, clinical protocol assistance, research grants and 10 years of market exclusivity following regulatory approval.
Woolsey Pharmaceuticals previously announced that the U.S. FDA had granted BRAVYL Orphan Drug Designation for ALS. A U.S. ODD provides certain benefits, including seven years market exclusivity upon regulatory approval.
ABOUT WOOLSEY PHARMACEUTICALS
New York-based Woolsey Pharmaceuticals is focused on the treatment of rare, neurodegenerative diseases. The company’s lead product is BRAVYL. The company currently has clinical trials for BRAVYL in wandering associated with dementia, Progressive Supranuclear Palsy (PSP) and Corticobasal Syndrome (CBS), two rare neurodegenerative disorders, as well as a study in ALS patients. BRAVYL is an investigational drug and is not approved to treat any disease or condition.